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Related Concept Videos

Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
Immunodeficiency Diseases01:25

Immunodeficiency Diseases

Immunodeficiency disorders are conditions in which the immune system's ability to fight infectious disease and cancer is compromised or entirely absent. The immune system comprises a complex network of cells, tissues, and organs that work together to protect the body from potentially harmful invaders. When this system is deficient or not functioning properly, it leaves the body susceptible to infections, diseases, or other complications.
There are three main causes of immunodeficiency disorders...
Tumor Immunotherapy01:27

Tumor Immunotherapy

Immunotherapy is a treatment that boosts or manipulates the immune system to fight diseases, including cancer. For instance, by stimulating an immune response through vaccinations against viruses that cause cancers, like hepatitis B virus and human papillomavirus, these diseases can be prevented. Nonetheless, some cancer cells can avoid the immune system due to their rapid mutation and division. The immune response to many cancers involves three phases: elimination, equilibrium, and escape.
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against specific...

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Related Experiment Video

Updated: May 17, 2026

Generation of Multivirus-specific T Cells to Prevent/treat Viral Infections after Allogeneic Hematopoietic Stem Cell Transplant
08:52

Generation of Multivirus-specific T Cells to Prevent/treat Viral Infections after Allogeneic Hematopoietic Stem Cell Transplant

Published on: May 27, 2011

Gene therapy for primary immunodeficiencies.

Eric Kildebeck1, Josh Checketts, Matthew Porteus

  • 1Department of Pediatrics, Stanford University School of Medicine, California 94305, USA.

Current Opinion in Pediatrics
|October 18, 2012
PubMed
Summary
This summary is machine-generated.

Gene therapy offers a promising alternative for primary immunodeficiencies (PIDs) by correcting genetic defects in hematopoietic stem cells. Recent advances in viral vectors and gene targeting technologies enhance safety and efficacy for treating these rare diseases.

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Related Experiment Videos

Last Updated: May 17, 2026

Generation of Multivirus-specific T Cells to Prevent/treat Viral Infections after Allogeneic Hematopoietic Stem Cell Transplant
08:52

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Published on: February 15, 2019

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Area of Science:

  • Immunology
  • Genetics
  • Molecular Biology

Background:

  • Primary immunodeficiencies (PIDs) are severe genetic disorders.
  • Hematopoietic stem cell transplantation is a potential treatment but limited by donor availability.
  • Gene therapy offers an alternative by genetically modifying a patient's own stem cells.

Purpose of the Study:

  • To review recent advancements in gene therapy for PIDs.
  • To highlight strategies for improving the safety of gene therapy.
  • To discuss the role of gene targeting technologies in PID treatment.

Main Methods:

  • Review of recent literature on gene therapy for PIDs.
  • Focus on novel viral vector designs (e.g., lentiviral vectors).
  • Examination of gene targeting technologies like engineered nucleases.

Main Results:

  • Improved safety profiles observed with new viral vectors in preclinical studies.
  • Clinical trials using advanced vectors are ongoing.
  • Gene targeting technologies have shown promise in correcting disease phenotypes for several PIDs in preclinical models.

Conclusions:

  • Advances in viral vector design and gene targeting offer safer and more effective gene therapy for PIDs.
  • These innovations have the potential to revolutionize PID treatment.
  • The goal is to provide effective treatment without significant patient risk.